Cardiac Amyloidosis in Asia

Q: What is the clinical evidence from this study?

Study design: Editorial. Population: Transthyretin amyloid cardiomyopathy (ATTR-CM) (n=32). Intervention: Vutrisiran vs. Placebo. Primary outcome: Composite of all-cause mortality and recurrent cardiovascular events (HR 0.20, 95% CI 0.04-0.93).

Q: What does this research mean for the field?

In East Asian patients with transthyretin amyloid cardiomyopathy, vutrisiran reduces the composite of all-cause mortality and recurrent cardiovascular events compared to placebo, showing clinical and pharmacodynamic effects consistent with the overall global trial. Novelty: ClaimNovelty.CONFIRMATORY. Consensus alignment: ConsensusAlignment.SUPPORTS_CONSENSUS.

Carolyn S.P. Lam; Li Shen; Chun Yuan Khoo

doi:10.1016/j.jacasi.2026.06.018

August 1, 2026JACC AsiaOpen Access

Cardiac Amyloidosis in Asia

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Key result

In a post hoc analysis of 32 East Asian patients with transthyretin amyloid cardiomyopathy, vutrisiran reduced the primary composite of all-cause mortality and recurrent cardiovascular events compared to placebo (HR 0.20).

Graphical abstract published by JACC Asia — Graphical abstract · Published by JACC Asia · CC BY-NC-ND

Why the study?

Does vutrisiran reduce all-cause mortality and recurrent cardiovascular events in East Asian patients with ATTR-CM?

Key Points

To assess the efficacy, pharmacodynamics, and regional applicability of the RNA-interference therapy vutrisiran among East Asian patients with transthyretin amyloid cardiomyopathy, and to evaluate the broader clinical landscape of cardiac amyloidosis across Asia.
Post hoc subgroup analysis of the randomized HELIOS-B trial evaluating East Asian patients (n = 32; 17 receiving vutrisiran and 15 receiving placebo) from Japan and South Korea followed through 42 months.
The primary composite endpoint evaluated all-cause mortality and recurrent cardiovascular events alongside pharmacodynamic monitoring of serum transthyretin reduction through 30 months.
Among East Asian patients with 8 total events, vutrisiran was associated with a reduction in the primary composite endpoint versus placebo (HR: 0.20, 95% CI: 0.04-0.93), compared with the global trial outcome (HR: 0.72, 95% CI: 0.56-0.93).
Vutrisiran achieved an approximately 89% reduction in serum transthyretin by month 30 in the East Asian subgroup, demonstrating pharmacodynamic knockdown consistent with the overall trial cohort.

Structured PICO

Does vutrisiran reduce all-cause mortality and recurrent cardiovascular events in East Asian patients with ATTR-CM?

P

Population

32 East Asian patients with transthyretin amyloid cardiomyopathy from Japan and South Korea, followed for up to 42 months.

I

Intervention

Vutrisiran (small-interfering RNA that silences hepatic transthyretin production)

C

Comparator

Placebo

O

Outcome

Composite of all-cause mortality and recurrent cardiovascular events through 42 monthscomposite

Main Result

Hazard Ratio: 0.2 (95% CI 0.04–0.93)

Limitations

A small subgroup of 32 patients cannot establish a region-specific treatment effect.
Results are inherently fragile with wide confidence intervals due to only 8 primary endpoints occurring.
Lack of adequately powered testing for region-by-treatment statistical interaction.
The East Asian subgroup was enriched for younger patients, NYHA class II status, wild-type disease, and less baseline tafamidis use, making it difficult to disentangle biology from case-mix.
Small subgroup size (n=32) cannot establish a region-specific treatment effect
Inherently fragile results with wide confidence intervals due to low event rates (8 primary endpoints)
Lack of adequately powered testing for region-by-treatment statistical interaction
Subgroup enriched for younger patients, NYHA class II, less baseline tafamidis use, and wild-type disease, making it difficult to disentangle biology from case-mix

Implication

Vutrisiran appears to provide consistent clinical and biological benefits in East Asian patients with ATTR-CM, though the evidence base in this population remains severely limited and requires expansion.

Abstract

[Figure: see text]

Discussion

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Cite This Study

Lam et al. (2026) conducted an editorial in Transthyretin amyloid cardiomyopathy (ATTR-CM) (n=32). Vutrisiran vs. Placebo was evaluated on Composite of all-cause mortality and recurrent cardiovascular events (HR 0.20, 95% CI 0.04-0.93). In a post hoc analysis of 32 East Asian patients with transthyretin amyloid cardiomyopathy, vutrisiran reduced the primary composite of all-cause mortality and recurrent cardiovascular events compared to placebo (HR 0.20).

synapsesocial.com/papers/6a8860c98dfe8b7ec5a54560 https://doi.org/10.1016/j.jacasi.2026.06.018

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Cardiac Amyloidosis in Asia

Key Points

Structured PICO

Main Result

Limitations

Implication

Abstract

Discussion

Cite This Study

Cardiac Amyloidosis in Asia

Key Points

Structured PICO

Main Result

Limitations

Implication

Abstract

Discussion

Cite This Study

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